Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment

Barbara E. Shapiro, Gregory M. Pastores, John G. Gianutsos, Cecile Paquet Luzy, Edwin H. Kolodny

Genetics in Medicine · 2009 · 106 citations · 35 references

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